Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.
Cardiopulmonary resuscitation (CPR) after cardiac arrest (CA) is often a cause of secondary neurological impairment, which results in considerable morbidity and mortality. Suppression of protein degradation of key blood-brain barrier (BBB) components after CPR could maintain the stability of the BBB function, and as such minimize secondary neurological damage and improve long-term prognosis after ischemia reperfusion injury.
Regor Pharmaceuticals Inc. has divulged phosphatidylinositol 3-kinase (PI3K) inhibitors reported to be useful for the treatment of cancer, congenital lipomatous overgrowth, vascular malformations, epidermal naevi, skeletal abnormalities and scoliosis, among others.
CVI Pharmaceuticals Ltd. has identified thyroid hormone receptor (THR)-β agonists reported to be useful for the treatment of atherosclerosis, hypercholesterolemia, hyperlipidemia, hepatic steatosis, thyroid cancer, obesity and hypothyroidism.
Hangzhou Polymed Biopharmaceuticals Inc. has synthesized crystalline forms of compounds acting as FLT3 (FLK2/STK1) and interleukin-1 receptor-associated kinase 4 (IRAK-4) inhibitors reported to be useful for the treatment of cancer, autoimmune diseases, cardiovascular, neurological and inflammatory disorders.
Antibody Therapeutics Inc. has disclosed integrin receptor antagonists reported to be useful for the treatment of pulmonary fibrosis, renal fibrosis and hepatic fibrosis.
Inherited retinal diseases (IRDs) are a group of monogenic disorders caused by mutations in more than 280 genes, and are characterized by progressive degeneration of photoreceptor cells in the retina.
Researchers from Shenyang Pharmaceutical University and affiliated organizations have detailed the discovery and preclinical characterization of novel reversible lysine-specific demethylase 1 (LSD1) inhibitors as candidates for acute myeloid leukemia (AML) therapy.